Opus Genetics Provides BEST1 Program Update with Detailed Timeline for OPGx-BEST1 Results

About this update from Opus Genetics, Inc.

Cohort 1 Topline Data Expected in the Second Week of September 2026 RESEARCH TRIANGLE PARK, N.C., July 09, 2026 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD) (the "Company", "Opus Genetics" or "Opus"), a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs), today provided updates on its ongoing OPGx-BEST1 Phase 1/2 clinical trial (BIRD-1) targeting BEST-1 associated IRDs. Opus expects to announce three-month topline data from Cohort 1 of the Phase 1/2 trial during the second week of September 2026, assuming all participants complete their assessments as scheduled. In addition, the Company plans to present the data at the annual EURETINA Congress taking place in Vienna, Austria from October 1 - 4, 2026. BIRD-1 is an adaptive, open-label, Phase 1/2 study evaluating the safety and efficacy of single-eye subretinal administration of OPGx-BEST1 in adult participants with Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB). The trial is designed as a dose escalation trial to evaluate two doses of OPGx-BEST1: 1.5E9 vg/eye (Cohort 1) and 4.5E9 vg/eye (Cohort 2). Enrollment in Cohort 1 was completed in May 2026 with five participants in the study, three with BVMD and two with ARB, who were carefully selected to meet the defined entry criteria. In the BVMD participants, the Company completed the added step of using an  in vitro  platform to confirm that each participant's disease mutation is amenable to gene augmentation. After the last participant has completed the Month 3 visit, the Independent Data Monitoring Committee (IDMC) will review all Cohort 1 data and determine next steps. In Cohort 1, the primary endpoint for evaluation is the safety and tolerability of OPGx-BEST1. In addition to safety measures, Opus will be also assessing a number of structural parameters including subretinal fluid as measured by Optical Coherence Tomography (OCT) scans. A reduction in subretinal fluid on OCT would suggest that OPGx-BEST1 has a biological effect demonstrating target engagement. A reduction trending towards 20% may be considered clinically meaningful, and these results, in addition to those on safety, are expected to be used to provide the rationale to advance the trial into Cohort 2 to optimize dose selection, per the trial protocol. Given the high level of patient interest in the trial, potential participants have already been identified to enroll in Cohort 2 if necessary. In the event that OPGx-BEST1 demonstrates a 100% reduction in fluid in the majority of patients in Cohort 1, the trial may be expanded into a potential pivotal trial. Opus expects to present the Month 3 data to the U.S. Food and Drug Administration (FDA) to align on the next steps for clinical development.

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